eprintid: 1481142 rev_number: 35 eprint_status: archive userid: 608 dir: disk0/01/48/11/42 datestamp: 2016-04-17 13:44:51 lastmod: 2021-12-13 01:33:00 status_changed: 2016-12-21 11:55:56 type: article metadata_visibility: show creators_name: Booth, C creators_name: Gaspar, HB creators_name: Thrasher, AJ title: Treating Immunodeficiency through HSC Gene Therapy ispublished: pub divisions: UCL divisions: B02 divisions: D13 divisions: G24 keywords: Science & Technology, Life Sciences & Biomedicine, Biochemistry & Molecular Biology, Cell Biology, Medicine, Research & Experimental, Research & Experimental Medicine, Chronic Granulomatous-disease, Leukocyte Adhesion Deficiency, Hematopoietic Stem-cells, Adenosine-deaminase-deficiency, Wiskott-aldrich Syndrome, Lentiviral Vector, Lymphoproliferative Disease, Murine Model, X-cgd, Mice note: Copyright © 2016. This manuscript version is published under a Creative Commons Attribution Non-commercial Non-derivative 4.0 International licence (CC BY-NC-ND 4.0). This licence allows you to share, copy, distribute and transmit the work for personal and non-commercial use providing author and publisher attribution is clearly stated. Further details about CC BY licences are available at http://creativecommons.org/licenses/by/4.0. Access may be initially restricted by the publisher. abstract: Haematopoietic stem cell (HSC) gene therapy has been successfully employed as a therapeutic option to treat specific inherited immune deficiencies, including severe combined immune deficiencies (SCID) over the past two decades. Initial clinical trials using first-generation gamma-retroviral vectors to transfer corrective DNA demonstrated clinical benefit for patients, but were associated with leukemogenesis in a number of cases. Safer vectors have since been developed, affording comparable efficacy with an improved biosafety profile. These vectors are now in Phase I/II clinical trials for a number of immune disorders with more preclinical studies underway. Targeted gene editing allowing precise DNA correction via platforms such as ZFNs, TALENs and CRISPR/Cas9 may now offer promising strategies to improve the safety and efficacy of gene therapy in the future. date: 2016-04 date_type: published publisher: ELSEVIER SCI LTD official_url: http://dx.doi.org/10.1016/j.molmed.2016.02.002 oa_status: green full_text_type: other language: eng primo: open primo_central: open_green article_type_text: Review verified: verified_manual elements_id: 1120523 doi: 10.1016/j.molmed.2016.02.002 lyricists_name: Booth, Claire lyricists_name: Gaspar, Hubert lyricists_name: Thrasher, Adrian lyricists_id: CABOO05 lyricists_id: HBGAS19 lyricists_id: ATHRA78 full_text_status: public publication: Trends In Molecular Medicine volume: 22 number: 4 pagerange: 317-327 pages: 11 issn: 1471-4914 citation: Booth, C; Gaspar, HB; Thrasher, AJ; (2016) Treating Immunodeficiency through HSC Gene Therapy. Trends In Molecular Medicine , 22 (4) pp. 317-327. 10.1016/j.molmed.2016.02.002 <https://doi.org/10.1016/j.molmed.2016.02.002>. Green open access document_url: https://discovery.ucl.ac.uk/id/eprint/1481142/1/Booth_Treating%20Immunodeficiency%20through%20HSC%20Gene%20Therapy.pdf document_url: https://discovery.ucl.ac.uk/id/eprint/1481142/2/Booth_Treating%20Immunodeficiency%20through%20HSC%20Gene%20Therapy%20Figure%201.pdf document_url: https://discovery.ucl.ac.uk/id/eprint/1481142/3/Booth_Treating%20Immunodeficiency%20through%20HSC%20Gene%20Therapy%20Figure%202.pdf